The US Food and Drug Administration announced on Friday that it is investigating a risk of neoplasia, which includes the formation of malignant tumors, associated with the gene therapy Zolgensma produced by Novartis.
Earlier that same day, the Swiss pharmaceutical company disclosed two specific cases where tumors in children showed evidence of being linked to the drug. Zolgensma is used to treat patients under the age of two who suffer from spinal muscular atrophy. Novartis stated it has received a total of nine reports of tumors in patients treated with Zolgensma since the therapy received approval in 2019.
The FDA maintained that the overall benefits of both Zolgensma and Novartis' other gene therapy, Itvisma, continue to outweigh potential risks for their approved uses. However, the agency is currently evaluating whether regulatory action is appropriate given the new risk. The FDA noted there have been no reported cases of neoplasia following the administration of Itvisma, which was approved last year for patients aged two and older.
Zolgensma functions by using a modified virus to deliver a working copy of a gene into cells. Novartis confirmed that in two of the recent cases, doctors found evidence that the carrier used in the treatment inserted its genetic material into the patients' own DNA. These findings suggest a possible link between the tumors and the one-time therapy, which Novartis confirmed has been administered to more than 6,000 children.
The investigation follows complaints from patient advocates who claimed Novartis was slow to share information regarding these cases. Novartis stated it notified health authorities in a timely and appropriate manner for both instances and noted that the potential risk of tumors was already included in the prescribing information for Zolgensma.
The patient advocacy organization SMA Europe has urged Novartis to provide information regarding what is known, what remains uncertain, and what the situation could mean for children who have already received the treatment. Novartis did not specify the locations of the two new cases in its statement.
